Freeline to present further FLT180a data at EAHAD
London, 21 January 2020 – Freeline, a biotechnology company focused on developing curative gene therapies for chronic systemic diseases, today announces it will be disclosing further data from its Phase 1/2 B-AMAZE trial investigating a novel gene therapy, FLT180a, for Haemophilia B on Friday 7 February 2020 at 14:00 – 15:00 pm CET at the 13th Annual Congress of the European Association for Haemophilia and Allied Disorder (EAHAD), The Hague, Netherlands. Previously, Freeline has disclosed 66- and 74-week data for the first cohort of two patients who were treated with the lowest study dose (4.5x1011vg/kg).
The Hot Topics session details have been published today and are available on the EAHAD website at https://eahadcongress.com/programme/programme/#friday.
Title: Phase 1/2 interim data from B-AMAZE study of adeno associated virus (AAV) gene therapy (FLT180a) confirms progress towards achieving Factor IX levels in the normal range for patients with severe or moderately severe haemophilia B
Presenter: Dr Pratima Chowdary
Session Date and Time: Friday 7 February 2020 at 14:00 – 15:00 pm CET
Freeline is a privately-held clinical-stage biotechnology company focused on AAV based gene therapy targeting the liver. Our vision is to create better lives for people suffering from chronic systemic diseases using the potential of gene therapy as a one-time curative treatment. Freeline is headquartered in the UK and has operations in Germany and the US.
About Haemophilia B
Haemophilia B is an inherited, X-linked disease characterized by spontaneous and prolonged bleeds in all tissues, including the joints, muscles and central nervous system. Haemophilia B is caused by mutations of the F9 gene encoding for coagulation factor IX (FIX) which plays an essential role in normal blood coagulation. Manifestations of Haemophilia can have lasting effects e.g., chronic arthropathy, life-threatening soft tissue bleeds, and CNS haemorrhages which can prove fatal. The life expectancy for Haemophilia B patients receiving treatment is 63-75 years dependent on severity of disease, and just 11 years for those without access to treatment.
Current treatment is aimed at preventing and treating bleeds through frequent intravenous infusions of Factor IX thus replacing the missing coagulation factor. Prophylactic use of FIX infusions reduces the number of bleeding episodes however due to the low FIX levels achieved and the peaks and troughs of FIX levels, the risk of development of arthropathy persist and patients are at continued risk of serious, even fatal bleeds e.g. because of trauma.